Igf2-tagging of gaa promotes full correction of murine pompe disease at a clinically relevant dosage of lentiviral gene therapy

HIGHLIGHTS

• who: Qiushi Liang from the Research Laboratory Sichuan University Center, Rotterdam GE, the Netherlands have published the research: IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy, in the Journal: (JOURNAL) of 27/Dec/2022
• what: The authors investigated the therapeutic efficacy of HSPC-mediated gene therapy using LV-IGF2.GAAco. This model reflects human Pompe disease caused by deficiency of GAA, resulting in generalized glycogen storage in various tissues, including skeletal muscle, the heart, and the brain.65,106-108 Age-matched FVB . . .
  

   

  

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